The number of subjects experiencing a Class II-V adverse event within 120 days of the start of treatment that is probably or definitely related to the study medication

The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 30 days post-treatment [ Time Frame: 30 days ] [ Designated as safety issue: No ]

The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 60 days post-treatment [ Time Frame: 60 days ] [ Designated as safety issue: No ]

The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 120 days post-treatment [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at 60 days [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in the Myasthenia Gravis Composite score at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in the Myasthenia Gravis Composite score at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in Manual Muscle Testing (MMT) score at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in Manual Muscle Testing (MMT) score at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in Quality of Life Assessment (SF-36) at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in Quality of Life Assessment (SF-36) at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in acetylcholine receptor antibody titre level at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in acetylcholine receptor antibody titre level at day 120 [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Change from baseline in prednisone dose at day 60 [ Time Frame: 60 days ] [ Designated as safety issue: No ]

Change from baseline in prednisone dose at day 120 Days [ Time Frame: 120 days ] [ Designated as safety issue: No ]

Participants will receive one dose of GM-CSF (5 µg/kg) by subcutaneous injection for ten (10) consecutive days. The first dose of GM-CSF will be administered by the subject or caregiver under the observation and direction of the study staff during the baseline visit. The subject or caregiver will administer subsequent injections at home.

Other Name: LEUKINE® (sargramostim)

Detailed Description:

Twelve patients aged 18-80 with symptomatic generalized autoimmune MG that are not being treated with medication that suppresses their immune system, other than prednisone, will enter the study at UIC over a two year period. The study will involve a screening visit and visits at baseline and at days 5, 15, 30, 45, 60, 90, and 120. The study drug, Leukine (GM-CFS), is given by injection. Subjects will give themselves one dose of GM-CSF every day for 10 days. Study visits will include muscle testing, immunologic studies and quality-of-life studies.

Eligibility

Ages Eligible for Study:

18 Years to 80 Years

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Must be between 18 and 80 years of age

Established diagnosis of myasthenia based on: the presence of fatigable weakness of ocular, oropharyngeal, and/or limb muscles AND the presence of abnormal acetylcholine receptor binding antibodies ≥ 0.4 nmol/l.

Patients of childbearing potential must agree to use a medically acceptable form of contraception defined by consistent use of oral contraceptive medications or history of tubal ligation or men who are in sexual relationship with such women during and for at least 8 weeks following completion of the study.

Patient or designee must have the ability to self-inject investigational product

If thymectomized, the procedure must have been performed at least one year prior to screening.

Dose of current anticholinesterase drugs must be constant for 2 weeks prior to screening.

If taking prednisone, dose must be stable for ≥4 weeks prior to screening.

Must not have received plasm exchange or IVIG within 4 weeks of screening

Must not have received immuno-modulating agents within the 4 weeks of screening, including Azathioprine (Imuran), Cyclosporine (Sandimmune, Neoral), Mycophenolate mofetil (CellCept), GM-CSF (Filgrastim; Neupogen; pegfilgrastim, sargramostim), or any other chronic immunosuppressive agent

History of tuberculosis or evidence of latent tuberculosis (positive PPD skin test or a chest X-ray with evidence of tuberculosis)

vital capacity of less than 1.2 liters or on supplemental oxygen therapy.

Laboratories values which, at the time of the screening visit or at any time during the study that in the opinion of the Investigator would preclude participation in the study including: serum creatinine > 2.5 mg/dL, serum potassium < 3.5 mmol/L or > 5.5 mmol/L, serum aspartate transaminase (AST), alanine transaminase (ALT), or alkaline phosphatase (ALP)> 3 times the upper limit of normal, platelet count < 100,000/mm3, WBC count < 3,000 cells/mm3, Hemoglobin, hematocrit, or red blood cell count outside 30% of the upper or lower limits of normal

Receipt of a live vaccine within 3 months of screening

participation in another investigational drug study within 90 days of screening.

known hypersensitivity to GM-CSF or any of its components

Known HIV-positive status or known history of any other immuno-suppressing disease.

Any mycobacterial disease.

Active severe infections within 4 weeks before screening visit, or between the screening and baseline visits.

Untreated Lyme disease.

History of TB or TB exposure, chronic hepatitis B or hepatitis C, SLE, history of multiple sclerosis, transverse myelitis, optic neuritis or epilepsy.

History of recent alcohol or substance abuse (< 1 year)

Pregnant or lactating females

History of non-compliance with other therapies

abnormal mental status sufficient to exclude informed consent

History of any opportunistic infection - to include but not limited to Pneumocystis carinii, aspergillosis, histoplasmosis, or atypical mycobacterium

History of Sickle cell disease.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01555580